Life Science investor. Dabble in tech and defense investing. No tweets should be construed as investment advice. Put GOD 1st.
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ALT WOW! BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.
ALT Prime Medicine - a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced that the first patient has been dosed in the Company’s global Phase 1/2 clinical trial of PM577a, an investigational in vivo Prime Editor for H1069Q-mutated Wilson disease. The milestone marks the initiation of the first clinical study of an in vivo Prime Editing therapy from Prime Medicine, as well as the first clinical evaluation of the company’s liver franchise. The company also announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to PM577 for Wilson disease.
ALT Prime Medicine - a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced that the first patient has been dosed in the Company’s global Phase 1/2 clinical trial of PM577a, an investigational in vivo Prime Editor for H1069Q-mutated Wilson disease. The milestone marks the initiation of the first clinical study of an in vivo Prime Editing therapy from Prime Medicine, as well as the first clinical evaluation of the company’s liver franchise. The company also announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to PM577 for Wilson disease.
